FiercePharma18h
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
BioPharma Dive12h
PTC wins US approval of gene therapy for fatal enzyme disorder
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
Monthly Prescribing Reference12h
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval
The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...
FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever With Direct Administration In Brain
PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...
Pharmabiz5h
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...
Business Insider16h
PTC Therapeutics Gains Buy Rating Following FDA Approval of Groundbreaking Gene Therapy Kebilidi
The recent FDA approval of Kebilidi, a groundbreaking gene therapy for AADC deficiency, is a significant milestone for the company. It is the first gene therapy directly administered to the brain and ...
GlobalData on MSN18h
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene ...
PTC Therapeutics wins FDA nod for gene therapy
PTC Therapeutics (PTCT) stock on watch as company receives FDA approval for gene therapy Kebilidi for AADC deficiency. Read ...
FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients ...
pharmaphorum1d
PTC gets FDA okay for first brain-delivered gene therapy
Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during ...
Finanznachrichten1d
PTC Therapeutics, Inc.: PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
The gene therapy, which will be marketed in the United States with the brand name KEBILIDI (eladocagene exuparvovec-tneq), is indicated for the treatment of children and adults with AADC deficiency, ...
Reuters1d
US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder
The therapy, sold under the brand name Kebilidi, is a one-time treatment for children and adults with an ultra rare inherited ...